Salk researcher Samuel L. Pfaff has
been awarded a $15.6 million grant by the
California Institute for Regenerative Medicine
(CIRM) for translational research focusing on
developing a novel stem cell-based therapy for
Amyotrophic Lateral Sclerosis (ALS) – or Lou
Gehrig's Disease.

Pfaff, a professor in the Salk's Gene Expression
Laboratory and an investigator for the Howard
Hughes Medical Institute, will lead the group of
researchers who will work on the four-year project,
which marks the first CIRM funding explicitly
expected to result in FDA approval for clinical trials.

The research team will focus on astrocytes,
the star-shaped support cells that provide nutrients
for nearby motor neurons. Working with six
different lines of human embryonic stem cells
(hESC), the team will grow clinical-grade astrocyte
precursor cells and identify the line that is
best suited for implantation in laboratory models.

They hypothesize that the transplanted human
astrocyte precursors (hAP) will mature into
astrocytes in vivo and provide support for diseased
spinal motor neurons. Astrocytes are also
capable of clearing excess neurotoxic glutamate
and could thereby slow or halt the progression of
ALS by preventing motor neuron degeneration.

Once the astrocyte precursors are tested for
efficacy and safety to minimize the possibility
of tumorigenesis, the next step will be to move
forward with human clinical trials after approval
by the FDA, says Pfaff.

"This team grant is a natural fit for San Diego
because it capitalizes on the strength of neuromuscular
disease research from the local scientific
community," says Pfaff. "The standard has been
set very high on this project because we are aiming
to grow a safe population of astrocytes that can
be introduced into patients. Our success will be
measured by whether it will help extend the lives
of patients suffering from ALS."